Background Therapeutic options for paediatric inflammatory bowel disease (IBD) are limited, especially for younger children. Unlike in adults, vedolizumab and ustekinumab are not licensed for paediatric use in the UK. We aimed to understand the real-world access to, and use of, these therapies in the paediatric population.
Methods We surveyed UK IBD centres to assess the incident use of vedolizumab and ustekinumab from 1 January 2021 to 31 December 2021. We collected information on funding, dose escalations and therapeutic drug monitoring.
Results 18 of 21 centres responded, covering an estimated 5260 patients. One hundred and thirteen were started on vedolizumab, prescription incidence 2.2%, median prescriptions per centre was 4 (range 1–20). Considering ustekinumab, 73 patients were commenced, prescription incidence 1.4%. Median prescription per centre was 3.5 (range 1–13). Prescription rates at each centre were not predicted by patient number cared for at that centre (p=0.2). Dose escalation was common in vedolizumab (66.7% centres) and ustekinumab (55.5%).
Funding strategies varied substantially, and multiple funding sources were used; 12 of 18 centres (66.7%) reported funding through routine National Health Service (NHS) England/Scottish arrangements. There was local NHS trust funding in 8 of 18 centres (44.4%). Individual funding requests (IFRs) were used in 5 of 18 (27.8%), although IFRs are reserved for patients with unique additional characteristics. Four centres were unable to achieve funding in pre-pubescent children.
Conclusions There is widespread use of vedolizumab and ustekinumab across the UK, although practice is highly variable. Access to therapy appeared to differ substantially. There is a growing disparity between international guidelines and real-world practice. Establishing early and effective therapy in all patients remains a priority.
- Healthcare Disparities
Data availability statement
Data are available upon reasonable request.
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MK-HA and JJA are joint first authors.
Collaborators Kwang Yang Lee, Ewan Swann, Vikki Garrick, Franco Torrente, Protima Deb, Helen Doble, Rafeeq Muhammed, Thankam Paul, Tracy Coelho, Veena Zamvar, Priya Narula, Andrew Fagbemi, Hemant S Bhavsar.
Contributors MKHA, JJA and JK conceived the idea for the study and formulated the survey. The survey was distributed by JJA. All authors contributed data to the study by completing the online survey. Data were collated and analysed by JJA and MKHA. JJA and MKHA wrote the manuscript with help from JK. All authors were able to comment on the article prior to submission.
Funding JJA is funded by an NIHR clinical lectureship. This research received no specific grant from any funding agency in the public, commercial or not-for-profit sectors.
Competing interests None declared.
Provenance and peer review Not commissioned; externally peer reviewed.
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